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Drug Trials Snapshots: XPOVIO | FDA

Drug Trials Snapshots: XPOVIO .HOW TO USE THIS SNAPSHOTThe information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs. LIMITATIONS OF THIS SNAPSHOT:Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the XPOVIO Prescribing Information for complete information. XPOVIO (selinexor) (x-PO-Vee-O) Karyopharm Therapeutics Inc. Approval date: July 3, 2019 DRUG TRIALS SNAPSHOT SUMMARY:. What is the drug for?. XPOVIO is a drug used to treat a form of blood cancer called multiple myeloma. It is to be used in combination with dexamethasone (a type of corticosteroid) in patients with no other treatment options whose cancer came back after, or did not respond to, at least four previous treatments. How is this drug used?. XPOVIO is a tablet. Four tablets (80mg) are taken by mouth in combination with dexamethasone on days 1 and 3 of each week. The XPOVIO dose may be decreased according to a special schedule if the patient cannot tolerate the side effects. What are the benefits of this drug?. In the trial, 21 of 83 patients (25%) treated with XPOVIO in combination with dexamethasone experienced improvement in their disease that lasted about 4 months. XPOVIO was approved under FDA’s accelerated approval program, which provides earlier patient access to a promising new drug while the company continues to conduct clinical trials to confirm that the drug works well. MORE INFO.What are the benefits of this drug (results of trials used to assess efficacy)?. The efficacy of XPOVIO was evaluated by overall response rate (ORR), as assessed by an Independent Review Committee (IRC) based on the International Myeloma Working Group (IMWG) Uniform Response Criteria for Multiple Myeloma. Table 2. Overall Response as Assessed by the IRC per IMWG Criteria Response Trial (N=83) Overall Response Rate (ORR)a, n (%). 21 (25.3).95% CI. 16.4, 36.Stringent Complete Response (sCR). 1 (1).Complete Response (CR). 0.Very Good Partial Response (VGPR). 4 (5).Partial Response (PR). 16 (19). a Includes sCR + CR + VGPR + PR XPOVIO Prescribing Information Were there any differences in how well the drug worked in clinical trials among sex, race and age?. Sex: XPOVIO was similarly effective in men and women.. Race: The majority of patients were White. Differences in response to XPOVIO among different races could not be determined.. Age: XPOVIO was similarly effective in patients below and above 65 years of age.. MORE INFO.Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?. The table below summarizes subgroup analysis of ORR. Table 3. Subgroup Analysis of ORR for mITT Population Demographic Subgroup (N = 83) ORR, n (%); [95% CI] Sex. Men (N = 51). 12 (23.5); [12.8, 37.5]. . Women (N = 32). 9 (28.1); [13.8, 46.8].Race. White (N = 58). 15 (25.9); [15.3, 39]. . Black or African American (N = 13). 3 (23.1); [5, 53.8]. . Othera (N=12). 3 (25); [4.2, 83.3].Age. 18-64 years (N = 40). 10 (25); [12.7, 41.2]. . 65-74 years (N = 31). 7 (22.6); [9.6, 41.1]. . ≥ 75 years (N = 12). 4 (33.3); [9.9, 65.1].Region. US (N = 58). . Non-US (N = 25). 17 (29.3); [18.1, 42.7]. a Other includes “Other,” “Missing,” “Asian,” and “Hawaiian/Pacific Islander” Adapted from FDA Review What are the possible side effects?. XPOVIO may cause serious reactions including low blood platelet count (thrombocytopenia), low neutrophil count (neutropenia), infections, gastrointestinal and nervous toxicity and low blood sodium. The most common side effects of XPOVIO are low blood counts, nausea, fatigue, decreased appetite, decreased weight, diarrhea, vomiting, low blood sodium, constipation, shortness of breath, and upper respiratory tract infections. MORE INFO.What are the possible side effects (results of trials used to assess safety)?. The table below summarizes the adverse reactions. Table 4. Adverse Reactions with XPOVIO 80 mg and Dexamethasone 20 mg Administered Twice Weekly Adverse Reaction Any Grade (N = 202) n (%) Grade ≥3 (N = 202) n (%) Thrombocytopeniaa. 149 (74). 124 (61).Fatigueb. 147 (73). 44 (22).Nausea. 146 (72). 18 (9).Anemiac. 119 (59). 81 (40).Decreased appetite. 108 (53). 9 (4.5).Weight decreased. 95 (47). 1 (0.5).Diarrhea. 89 (44). 13 (6).Vomiting. 82 (41). 7 (3.5).Hyponatremia. 78 (39). 44 (22).Neutropeniad. 68 (34). 43 (21).Leukopenia. 57 (28). 23 (11).Constipation. 50 (25). 3 (1.5).Dyspneae. 48 (24). 7 (3.5)k.Upper respiratory tract infectionf. 42 (21). 6 (3).Coughg. 33 (16). 0.Mental status changesh. 33 (16). 14 (7).Pyrexia. 32 (16). 1 (0.5).Hyperglycemia. 31 (15). 15 (7).Dizziness. 30 (15). 0.Insomnia. 30 (15). 4 (2).Lymphopenia. 30 (15). 20 (10).Dehydration. 28 (14). 7 (3.5).Hypercreatininemiai. 28 (14). 4 (2).Pneumoniaj. 26 (13). 18 (9)k.Epistaxis. 25 (12). 1 (0.5).Hypokalemia. 25 (12). 7 (3.5).Dysgeusia. 22 (11). 0.Vision blurred. 21 (10). 1 (0.5).Headache. 20 (10). 0. a. Thrombocytopenia includes thrombocytopenia and platelet count decreased. b. Fatigue includes fatigue and asthenia. c. Anemia includes anemia and hematocrit decreased. d. Neutropenia includes neutropenia and neutrophil count decreased. e. Dyspnea includes dyspnea, dyspnea exertional, and dyspnea at rest. f. Upper respiratory tract infection includes upper respiratory tract infection, respiratory tract infection, pharyngitis, nasopharyngitis, bronchitis, bronchiolitis,respiratory syncytial virus infection, parainfluenza virus infection, rhinitis, rhinovirus infection, and adenovirus infection. g. Cough includes cough, productive cough, and upper‐airway cough syndrome. h. Mental status changes includes mental status changes, confusional state, and delirium. i. Hypercreatininemia includes hypercreatininemia and hypercreatinemia. j. Pneumonia includes pneumonia, atypical pneumonia, lung infection, lower respiratory tract infection, pneumocystis jirovecii pneumonia, pneumonia aspiration, pneumonia influenzal, and pneumonia viral. k. Includes fatal event XPOVIO Prescribing Information Were there any differences in side effects among sex, race and age?. Sex: The occurrence of side effects was similar in men and women.. Race: The occurrence of side effects between White and Black or African American patients was similar. Differences in side effects among other races could not be determined because of the small number of patients in those races.. Age: The occurence of overall side effects was similar in patients below and above 65 years of age. Certain side effects—called serious adverse events1 --were seen more frequently in patients age 75 and above compared to those below the age of 75.. 1 Serious adverse event is defined as any event that results in one of the following: death, life-threatening event, required hospitalization or extended a current hospital stay, persistent or significant disability/incapacity, or congenital anomaly or birth defect. MORE INFO.Were there any differences in side effects of the clinical trials among sex, race, and age groups?. The tables below summarize adverse events (AE) by subgroups. Table 5. Summary of Adverse Reactions by Sex AE Category Men N= 108 n (%) Women N = 94 n (%) Any TEAE. 108 (100). 94 (100).Any Grade 3 or 4 TEAE. 100 (92.6). 90 (95.7).Serious TEAE. 63 (58.3). 55 (58.5).Death. 11 (10.2). 7 (7.4). Table 6. Summary of Adverse Reactions by Race AE Category White N= 148 n (%) Black or African American N = 35 n (%) Any TEAE. 148 (100). 35 (100).Any Grade 3 or 4 TEAE. 138 (93.2). 33 (94.3).Serious TEAE. 87 (58.8). 21 (60).Death. 13 (8.8). 2 (5.7). Table 7. Summary of Adverse Reactions by Age AE Category <65 years N=105 n (%) ≥65 years N=97 n(%) ≥75 years N=23 N (%) Any TEAE. 105 (100). 97(100). 23 (100).Any Grade 3 or 4 TEAE. 101 (96.2). 89 (91.8). 19 (82.6).Serious TEAE. 60 (57.1). 58 (59.8). 16 (69.6).Death. 9 (8.6). 9 (9.8). 4 (17.4). TEAE=treatment-emergent adverse event Clinical Trial Data WHO WAS IN THE CLINICAL TRIALS?. Who participated in the clinical trials?. The FDA approved XPOVIO based on evidence from a clinical trial (NCT02336815) of 202 patients with multiple myeloma whose disease came back after, or did not respond to, previous treatments. The trial was conducted at 58 sites in US and Europe. Presented below are all 202 patients who provided data for evaluation of side effects (called the safety population). Some of these patients (83) also provided data for evaluation of the benefits. Demographics of these patients are presented in Table 9 under MORE INFO. Figure 1 summarizes how many men and women were in the clinical trial. Figure 1. Baseline Demographics by Sex (safety population)FDA Review Figure 2 and Table 1 summarize the percentage of patients by race enrolled in the clinical trial. Figure 2. Baseline Demographics by Race (safety population)FDA Review Table 1. Demographics of Efficacy Trials by Race Race Number of Patients Percentage White. 148. 73.Black or African American. 35. 17.Asian. 2. 1.Native Hawaiian or other Pacific Islander. 1. less than 1.Other. 10. 5.Missing. 6. 3. FDA Review Figure 3 summarizes the percentage of patients by age group enrolled in the clinical trial. Figure 3. Baseline Demographics by Age (safety population)FDA Review MORE INFO.Who participated in the trials?. The tables below summarize safety and efficacy populations, respectively. Table 8. Demographics of Patients in the Clinical Trial (safety population) Demographic Subgroup   XPOVIO+ Dexamethasone N=202 Sex, n (%). Men. 108 (54). . Women. 94 (47).Race, n (%). White. 148 (73). . Black or African American. 35 (17). . Asian. 2 (1). . Native Hawaiian or other Pacific Islander. 1 (1). . Other. 10 (5). . Missing. 6 (3).Age Group, n (%). Age (median range). 64 (34-85). . <65 years. 105 (52). . >=65 years. 97 (48).Ethnicity, n(%). Hispanic or Latino. 11 (5). . Not Hispanic or Latino. 172 (85). . Not reported. 19 (9).Geographic region. Europe. 39 (19). . USA. 163 (81). Table 9. Demographics of Patients in the Clinical Trial (efficacy population) Demographic Parameter   XPOVIO (N = 83) Sex, n (%). Men. 51 (61). . Women. 32 (39).Race, n (%). White. 58 (70). . Black or African American. 13 (16). . Asian. 2 (2). . Native Hawaiian or other Pacific Islander. 1 (1). . Other. 6 (7). . Missing. 3 (4).Age category, n (%). Median age, years (range). 65 (40, 86). . <65 years. 40 (48). . 65 – 75 years. 31 (37). . >75 years. 12 (15). FDA Review How were the trials designed?. All patients in the trial received XPOVIO in combination with dexamethasone twice a week until the disease progressed or the side effects became too toxic. The benefit of XPOVIO was measured by the proportion of patients that achieved improvement (overall response rate or ORR). MORE INFO.How were the trials designed?. The efficacy and safety of XPOVIO in combination with dexamethasone were evaluated in a multicenter, single-arm, open-label trial of patients with relapsed refractory multiple myeloma who had previously received 3 or more anti-myeloma treatment regimens, and whose disease no longer responded to at least one type of proteasome inhibitor, one type of an immunomodulatory agent, and one type of an anti-CD38 monoclonal antibody. Treatment was administered twice weekly until disease progression or unacceptable toxicity. The primary endpoint was ORR, defined as the proportion of patients with a PR or better, as assessed by an IRC based on the IMWG Uniform Response Criteria for Multiple Myeloma. GLOSSARY. CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments. COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested. EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial. PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups. PRESCRIBING INFORMATION. Back to Drug Trials Snapshots.